Systemic delivery of MicroRNA using recombinant adeno-associated virus serotype 9 to treat neuromuscular diseases in rodents by Pourshafie, N, Lee, P, Chen, K, Harmison, G, Bott, L, Fischbeck, K, Rinaldi, C

Gene therapy with AR isoform 2 rescues spinal and bulbar muscular atrophy phenotype by modulating AR transcriptional activity by Lim, WF, Forouhan, M, Roberts, TC, Dabney, J, Ellerington, R, Speciale, AA, Manzano, R, Lieto, M, Sangha, G, Banerjee, S, Conceição, M, Cravo, L, Biscans, A, Roux, L, Pourshafie, N, Grunseich, C, Duguez, S, Khvorova, A, Pennuto, M, Cortes, CJ, La Spada, AR, Fischbeck, KH, Wood, MJA, Rinaldi, C